Using focused ultrasound to deliver GCase for Parkinson's disease
A Phase I/II Study for Bilateral Putamenal Delivery of Recombinant Glucocerebrosidase in Patients With Parkinson's Disease Using MR-guided Focused Ultrasound Induced Opening of the Blood-brain Barrier
This study is testing if using focused ultrasound to deliver a treatment directly into the brain can help people with Parkinson's disease feel better.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 14 (estimated) |
| Ages | 35 Years to 80 Years |
| Sex | All |
| Sponsor | InSightec Industry-sponsored |
| Drugs / interventions | bevacizumab |
| Locations | 1 site (Toronto, Ontario) |
| Trial ID | NCT05565443 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and feasibility of delivering GCase directly into the brain using MR-guided focused ultrasound in patients with Parkinson's disease. The study consists of two arms, one for patients with GBA mutations and another for idiopathic Parkinson's disease, with a total of fourteen participants. Each participant will receive a series of GCase treatments over a period of time, followed by a year of monitoring to assess outcomes. The approach is based on promising results from animal models showing potential benefits in improving Parkinson's disease symptoms.
Who should consider this trial
Good fit: Ideal candidates include men and women aged 35 to 80 with a diagnosis of Parkinson's disease and specific genetic criteria related to GBA mutations.
Not a fit: Patients who are pregnant, have contraindications to MRI or ultrasound contrast agents, or have poorly controlled diabetes may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients with Parkinson's disease, potentially improving their symptoms and quality of life.
How similar studies have performed: While the use of focused ultrasound for drug delivery is a novel approach, similar studies have shown promise in other neurological conditions, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Men or women between age 35 and 80 years, inclusive. * Able and willing to give informed consent. * Diagnosis of PD satisfying MDS Clinical Diagnostic Criteria for PD * At least 2 years from initial diagnosis * Hoehn and Yahr Stage 1-3 on PD medication * Dopaminergic deficit by a positive DAT SPECT scan * On stable regimen of PD medications for at least 90 days prior to the study * American Society of Anesthesiologists (ASA) score 1-3 * Harbor at least one GBA mutation if enrolled in the GBA PD arm or two wild-type GBA alleles if enrolled in the idiopathic PD arm Exclusion Criteria: * Positive pregnancy test (for pre-menopausal women). * Contraindication to DEFINITY ultrasound contrast agent or perflutren (e.g. hypersensitivity, known left or bidirectional cardiac shunt) * Contraindication to MRI or gadolinium-DTPA (e.g. incompatible device, hypersensitivity) * Insulin-dependent diabetes mellitus that is not well-controlled or that in the Investigator's opinion precludes participation in the study. * Severely impaired renal function with estimated glomerular filtration rate \<30 mL/min/1.73m2 and/or on dialysis. * Currently participating in another clinical therapeutic trial * Patient receiving bevacizumab (Avastin) therapy * Subjects with evidence of cranial or systemic infection. * Cerebral or systemic vasculopathy. * Documented cerebral infarction within the past 12 months or TIA in the past 1 month. * Contraindication to GCase enzyme therapy, specifically previous hypersensitivity reaction to GCase enzyme therapy * Parkinsonism plus symptoms, secondary parkinsonism * Previous neurosurgical procedure for PD
Where this trial is running
Toronto, Ontario
- Sunybrook Research Institute — Toronto, Ontario, Canada (Recruiting)
Study contacts
- Study coordinator: Alyssa Voelker-Christy
- Email: alyssav@insightec.com
- Phone: +1-817-948-5219
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.